PRESS RELEASE - Downingtown, PA – August 16, 2022 – The Friedreich’s Ataxia Research Alliance (FARA), a leading patient advocacy organization dedicated to developing treatments and cures for Friedreich’s Ataxia (FA), announced today that data from a FARA-sponsored natural history study – the FA Clinical Outcomes Measures Study (FACOMS) -- has helped provide evidence and understanding of the treatment effect of Omaveloxolone, a potential treatment currently under review for approval by the Food and Drug Administration (FDA).
FACOMS is a multicenter natural history study that aims to understand the clinical symptoms, progression, and long-term outcomes for individuals with FA. Initiated by a core group of clinical researchers and supported by FARA, the FACOMS natural history study is the largest, most diverse source of longitudinal clinical data in FA. FACOMS has facilitated the development of clinical outcome assessments being used in clinical trials, such as the modified Friedreich’s Ataxia Ratings Scale (mFARS), which assesses neurological function in FA patients.
The newly published study using FACOMS data is entitled Direct utility of natural history data in analysis of clinical trials: Propensity match-based analysis of Omaveloxolone in Friedreich ataxia using the FACOMS dataset, and is available via the preprint repository for health sciences- medRXiv https://medrxiv.org/cgi/content/short/2022.08.12.22278684v1. The Propensity match-based analysis of Omaveloxolone in FA using the FACOMS dataset compares 136 individuals who received Omaveloxolone (Omav) for FA in an open label extension study for an average of three years to well-matched individuals in FACOMS. The primary endpoint, change from baseline in mFARS score at year three, showed a 55% slowing in disease progression as measured by mFARS. The matched FACOMS cohort progressed by 6.6 mFARS points over three years and individuals in the MOXIe extension study (treated with Omav) progressed only 3.0 points (difference =-3.6 points; p=0.0001).
Jennifer Farmer, Chief Executive Officer of FARA, states, “This publication demonstrates the power of the natural history study in providing critical data to support clinical trials and shaving time off drug development. FARA is proud to have invested in and fostered this research. We are hopeful that this study will provide confirmatory evidence of the treatment effect of Omav observed in the MOXIe Part 2 study and can support an FDA decision to approve Omav for FA. In a progressive neuromuscular disease, we do not have the luxury of time and must operate with efficiency and urgency.”
Ron Bartek, President and Co- Founder of FARA adds, “Behind these data are more than 20 years of dedicated time from FA families, investigators throughout our Collaborative Clinical Research Network in FA, as well as generous contributions from donors to fund the work. The average distance a family travels to participate in a natural history visit is over 500 miles. We are grateful to all FA families for their long-standing commitment to research progress. We are beyond proud and excited to be a part of helping to demonstrate Omav can have a meaningful impact on the disease progression and hope that all FA community members who want access to Omav will have it soon.”